Building awareness and communities to support and find treatments for Rare Diseases and Genetic Conditions
On the last day of the World Orphan Drug Congress, John Crowley, Chairman and CEO, Amicus Therapeutics provided an inspiring overview of how the Fifth Prescription Drug User Fee Act (PDUFA V) and new government policies may affect the orphan drug industry. From a historical [...]
As a politician, one of the worst things you can be accused of is being a flip-flopper, a person who changes their mind on one or more issues. Another way of looking at this character trait is to say one has thoughtfully wrestled with an issue and changed [...]
As mandated by the Food and Drug Administration Safety and Innovation Act (FDASIA), the FDA just posted a draft of its “Structured Approach to Benefit-Risk Assessment in Drug Regulatory Decision Making.” There is a 60-day public comment period on the draft, and any individual or [...]
Reposted from The Catalyst. Written by Stephanie Fischer. While 400 therapies for rare diseases are worth celebrating, there is a lot more to learn about rare diseases in order to better diagnose and treat them. Many are inherited and caused by gene mutations, and new rare [...]
The Orphan Drug Act (“ODA”), which President Ronald Reagan signed into law on January 4, 1983, turned 30 years old in January, 2013. The milestone came just days after the death of actor Jack Klugman, who has been credited as being an important driving force behind the [...]
In their recent Advocacy Alert, the National Organization for Rare Disorders (NORD) encourages patient organization representatives to attend the following announcement: NORD President and CEO Peter L. Saltonstall to Speak at Capitol Hill Event Michael J. Astrue, Commissioner of Social Security, will announce the expansion [...]
The election is over, and the status-quo remains. Republicans remain in control of the House of Representatives, while Democrats retain control of the Senate and White House. When Congress returns to work next week for the lame duck session, they must act quickly to protect [...]
